お知らせ • Jun 07
Vertex Pharmaceuticals Incorporated Presents New Data on Benefits of ALYFTREK and Importance of Achieving Lower Sweat Chloride Levels at the European Cystic Fibrosis Conference
Vertex Pharmaceuticals Incorporated announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK®? (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data were presented at this year's European Cystic Fibrosis Society's (ECFS) 48th European Cystic Fibrosis Conference held June 4-7, 2025, in Milan, Italy. During the conference, the Company presented data from a pooled analysis across CFTR modulators, including ALYFTREK, which demonstrate that reduction in sweat chloride (SwCl), and therefore greater restoration of CFTR function, is associated with improved outcomes in people with cystic fibrosis (CF). For all clinical outcomes in the study, SwCl levels below 60 mmol/L were associated with greater benefit including better and more stable lung function, fewer pulmonary exacerbations, better nutritional status and better quality of life. SwCl levels below 30 mmol/L generally demonstrated greater numerical benefit than all other groups, with confidence intervals that overlapped with the 30 to About Cystic Fibrosis. Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be protective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. TRIKAFTA is indicated for the treatment of CF in patients aged 2 years and older who have at least one F508 Del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of the indicated mutation(s) for the respective product. Elevated transaminases have been observed in patients treated with ALYFTREK. Cases of liver failure leading to transplantation and death have been reported in both clinical trials and the postmarketing setting in patients with and without a history of liver disease taking TRIKAFTA, a fixed-dose combination drug containing elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA), the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA. Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter.